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Deferiprone in Parkinson’s Disease 

Compared with placebo, the iron chelator deferiprone produced significantly worse outcomes among patients with early Parkinson’s disease, a 36-week study shows. Prior research had indicated that chelation might be beneficial for reducing nigrostriatal iron content and thereby targeting a key pathophysiologic mechanism thought to be involved in Parkinson’s disease.

The multicenter, phase 2, randomized, double-blind trial included 372 participants with newly diagnosed Parkinson’s disease who had never received levodopa. They were randomized to oral deferiprone 15 mg/kg twice daily or matched placebo for 36 weeks; dopaminergic therapy was withheld unless necessary for symptom control.

Based on a primary outcome of the change in the total score on the Movement Disorder Society–sponsored revision of the Unified Parkinson’s Disease Rating Scale (MDS-UPDRS; range, 0 to 260, with higher scores indicating more severe impairment) at 36 weeks, the study showed: “Progression of symptoms led to the initiation of dopaminergic therapy in 22.0% of the participants in the deferiprone group and 2.7% of those in the placebo group. The mean MDS-UPDRS total score at baseline was 34.3 in the deferiprone group and 33.2 in the placebo group and increased (worsened) by 15.6 points and 6.3 points, respectively (difference, 9.3 points; 95% confidence interval, 6.3 to 12.2; P <0.001). Nigrostriatal iron content decreased more in the deferiprone group than in the placebo group. The main serious adverse events with deferiprone were agranulocytosis in 2 participants and neutropenia in 3 participants.”

Editorial: “The FAIRPARK-II trial provides evidence that deferiprone does not slow progression in Parkinson’s disease, but the data from this trial could nevertheless stimulate studies to clarify the role of iron in the pathogenesis of Parkinson’s disease,” editorialists write. “However, the findings raise cautions about iron chelation as a therapeutic intervention in neurodegenerative disorders.”

Source: New England Journal of Medicine